IL275617B2 - cationic lipids - Google Patents

cationic lipids

Info

Publication number
IL275617B2
IL275617B2 IL275617A IL27561720A IL275617B2 IL 275617 B2 IL275617 B2 IL 275617B2 IL 275617 A IL275617 A IL 275617A IL 27561720 A IL27561720 A IL 27561720A IL 275617 B2 IL275617 B2 IL 275617B2
Authority
IL
Israel
Prior art keywords
cationic lipids
lipids
cationic
Prior art date
Application number
IL275617A
Other languages
English (en)
Hebrew (he)
Other versions
IL275617B1 (en
IL275617A (en
Original Assignee
Takeda Pharmaceuticals Co
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Takeda Pharmaceuticals Co filed Critical Takeda Pharmaceuticals Co
Publication of IL275617A publication Critical patent/IL275617A/en
Publication of IL275617B1 publication Critical patent/IL275617B1/en
Publication of IL275617B2 publication Critical patent/IL275617B2/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C69/00Esters of carboxylic acids; Esters of carbonic or haloformic acids
    • C07C69/52Esters of acyclic unsaturated carboxylic acids having the esterified carboxyl group bound to an acyclic carbon atom
    • C07C69/533Monocarboxylic acid esters having only one carbon-to-carbon double bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1617Organic compounds, e.g. phospholipids, fats
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
IL275617A 2017-12-28 2018-12-27 cationic lipids IL275617B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2017254667 2017-12-28
PCT/JP2018/048054 WO2019131839A1 (ja) 2017-12-28 2018-12-27 カチオン性脂質

Publications (3)

Publication Number Publication Date
IL275617A IL275617A (en) 2020-08-31
IL275617B1 IL275617B1 (en) 2023-07-01
IL275617B2 true IL275617B2 (en) 2023-11-01

Family

ID=67063849

Family Applications (1)

Application Number Title Priority Date Filing Date
IL275617A IL275617B2 (en) 2017-12-28 2018-12-27 cationic lipids

Country Status (17)

Country Link
US (2) US20200331841A1 (pl)
EP (1) EP3733641B1 (pl)
JP (1) JP7211973B2 (pl)
KR (1) KR102589314B1 (pl)
CN (1) CN111511713B (pl)
AU (1) AU2018397957B2 (pl)
CA (1) CA3086824A1 (pl)
DK (1) DK3733641T3 (pl)
ES (1) ES2978261T3 (pl)
FI (1) FI3733641T3 (pl)
IL (1) IL275617B2 (pl)
MX (1) MX2020006800A (pl)
PL (1) PL3733641T3 (pl)
PT (1) PT3733641T (pl)
SG (1) SG11202006154UA (pl)
TW (1) TWI802627B (pl)
WO (1) WO2019131839A1 (pl)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
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MX2020006800A (es) 2017-12-28 2020-09-03 Takeda Pharmaceuticals Co Lipidos cationicos.
FI3835287T3 (fi) * 2018-08-10 2024-12-03 Takeda Pharmaceuticals Co Kationinen lipidi
EP3842538A4 (en) 2018-08-10 2022-06-08 Kyoto University METHOD OF TRANSFECTION INTO CARDIOMYOCYTES USING CATIONIC LIPID
CN119876276A (zh) 2018-10-18 2025-04-25 武田药品工业株式会社 T细胞的活化/增殖方法
WO2022045334A1 (ja) 2020-08-31 2022-03-03 武田薬品工業株式会社 推論装置、推論方法、推論プログラム、モデル生成方法、推論サービス提供システム、推論サービス提供方法及び推論サービス提供プログラム
KR20230118715A (ko) 2020-10-14 2023-08-11 조지 메이슨 리서치 파운데이션 인코포레이티드 이온화 가능 지질 및 이의 제조 및 사용 방법
AR127312A1 (es) 2021-10-08 2024-01-10 Suzhou Abogen Biosciences Co Ltd Compuestos lipídicos ycomposiciones de nanopartículas lipídicas
MX2024005608A (es) 2021-11-10 2024-05-22 Takeda Pharmaceuticals Co Lipido cationico.
US20250191703A1 (en) 2022-02-28 2025-06-12 Takeda Pharmaceutical Company Limited Inference device, generation device, inference program, and generation program
WO2023250197A2 (en) * 2022-06-23 2023-12-28 Turn Biotechnologies, Inc. Lipid structures and compositions comprising same
WO2024121814A1 (en) 2022-12-09 2024-06-13 Takeda Pharmaceutical Company Limited Modified immunomodulators
EP4687990A1 (en) 2023-03-30 2026-02-11 Ose Immunotherapeutics Method of synthesis of targeted lipid nanoparticle and uses thereof
JP2026512425A (ja) 2023-03-30 2026-04-16 オーエスイー・イミュノセラピューティクス 免疫細胞増強分子の発現のための活性化免疫細胞に標的化された脂質ベースのナノ粒子及びその使用
AR132276A1 (es) 2023-04-07 2025-06-11 Takeda Pharmaceuticals Co Complejo de conjugación
WO2024236361A1 (en) 2023-05-15 2024-11-21 Takeda Pharmaceutical Company Limited Compositions and methods for delivery of nucleic acids to cells
WO2024236504A1 (en) 2023-05-15 2024-11-21 Takeda Pharmaceutical Company Limited Sequences and methods for delivery of dna and rna
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
CN121620589A (zh) 2023-08-01 2026-03-06 法国居里学院 包含编码环gmp-amp合酶的核酸序列的纳米粒子
WO2025133115A1 (en) 2023-12-21 2025-06-26 Ose Immunotherapeutics Lipid-based nanoparticles comprising il-35
JP7836475B1 (ja) 2024-03-05 2026-03-26 武田薬品工業株式会社 Unc13aのスプライシングモジュレーションのための組成物および方法
WO2026068705A1 (en) 2024-09-26 2026-04-02 Ose Immunotherapeutics Lipid-based nanoparticles comprising non-glycosylated fc domains and uses thereof
WO2026093414A1 (en) 2024-10-30 2026-05-07 Ose Immunotherapeutics Lipid-based nanoparticles comprising membrane il-35

Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2003102150A2 (en) * 2002-05-31 2003-12-11 Genteric, Inc. Multi-functional polyamines for delivery of biologically-active polynucleotides

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US7223887B2 (en) 2001-12-18 2007-05-29 The University Of British Columbia Multivalent cationic lipids and methods of using same in the production of lipid particles
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
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CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
US9408914B2 (en) 2010-04-28 2016-08-09 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
JP2013544488A (ja) 2010-08-30 2013-12-19 エフ.ホフマン−ラ ロシュ アーゲー テトラネクチン−アポリポタンパク質a−i脂質粒子を産生するための方法、脂質粒子自体、及びその使用
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ES3029138T3 (en) 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
BR112016013213A2 (pt) 2013-12-12 2017-12-05 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas
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KR101882634B1 (ko) 2014-08-07 2018-07-26 다케다 야쿠힝 고교 가부시키가이샤 양이온성 지질
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CN104873976B (zh) 2015-05-08 2016-04-20 厦门成坤生物技术有限公司 一类阳离子类脂化合物的应用
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
AU2016344609B2 (en) 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
MX2020006843A (es) 2017-12-27 2020-09-03 Takeda Pharmaceuticals Co Nanoparticula de lipido que contiene acido nucleico y uso de la misma.
TWI825057B (zh) 2017-12-28 2023-12-11 國立大學法人京都大學 標的基因改變用組成物
MX2020006800A (es) 2017-12-28 2020-09-03 Takeda Pharmaceuticals Co Lipidos cationicos.

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003102150A2 (en) * 2002-05-31 2003-12-11 Genteric, Inc. Multi-functional polyamines for delivery of biologically-active polynucleotides

Also Published As

Publication number Publication date
SG11202006154UA (en) 2020-07-29
KR102589314B1 (ko) 2023-10-13
MX2020006800A (es) 2020-09-03
EP3733641A4 (en) 2021-11-03
ES2978261T3 (es) 2024-09-09
US20200331841A1 (en) 2020-10-22
BR112020012424A2 (pt) 2020-11-24
AU2018397957A1 (en) 2020-07-09
JPWO2019131839A1 (ja) 2021-01-21
IL275617B1 (en) 2023-07-01
CA3086824A1 (en) 2019-07-04
CN111511713B (zh) 2024-01-02
PL3733641T3 (pl) 2024-07-15
AU2018397957B2 (en) 2024-05-23
JP7211973B2 (ja) 2023-01-24
TWI802627B (zh) 2023-05-21
US11993570B2 (en) 2024-05-28
EP3733641A1 (en) 2020-11-04
CN111511713A (zh) 2020-08-07
DK3733641T3 (da) 2024-07-22
PT3733641T (pt) 2024-05-07
EP3733641B1 (en) 2024-04-10
US20230257338A1 (en) 2023-08-17
RU2020124751A (ru) 2022-01-28
KR20200103679A (ko) 2020-09-02
RU2020124751A3 (pl) 2022-02-22
WO2019131839A1 (ja) 2019-07-04
FI3733641T3 (fi) 2024-06-28
TW201932446A (zh) 2019-08-16
IL275617A (en) 2020-08-31

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